casimersen
Sign in to saveAlso known as Amondys 45, SRP-4045
Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO). Duchenne muscular dystrophy is a rare disease that primarily affects boys. It is caused by low levels of a muscle protein called dystrophin. The lack of dystrophin causes progressive muscle weakness and premature death.
Medication
- Generic name
- Casimersen
- Brand names
- Amondys 45
- Drug class
- Antisense Oligonucleotide
- Route
- Intravenous
- Type
- Human Prescription Drug
- Manufacturer
- Sarepta Therapeutics, Inc.
Indications: 1 INDICATIONS AND USAGE AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin producti… (per FDA label)
Clinical Trials
3 registered- PHASE3TERMINATEDAn Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular DystrophySarepta Therapeutics, Inc. · NCT03532542
- PHASE3COMPLETEDStudy of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)Sarepta Therapeutics, Inc. · NCT02500381
- ENROLLING_BY_INVITATIONA Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical PracticeSarepta Therapeutics, Inc. · NCT06606340
~3 min read
Encyclopedic overview
7 sectionsContents
- Medical uses
- Adverse effects
- Pharmacology
- Pharmacodynamics
- History
- References
- External links
Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO). Duchenne muscular dystrophy is a rare disease that primarily affects boys. It is caused by low levels of a muscle protein called dystrophin. The lack of dystrophin causes progressive muscle weakness and premature death.
The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.
Excerpted from Wikipedia’s “casimersen” article, available under the CC BY-SA 4.0 licence.